ZURICH (Reuters) – Swiss drugmaker Novartis on Wednesday said it had won European Union approval for Rydapt to be used against a mutated form of acute myeloid leukemia (AML) and other rare diseases, adding to U.S. approvals it secured in April.

Source: Reuters: HealthCategory: Consumer Health News Tags: healthNews Source Type: news

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Publication date: Available online 3 May 2018Source: Oral Surgery, Oral Medicine, Oral Pathology and Oral RadiologyAuthor(s): Fang Yu, Ke Sun, Zhaoming WangBlastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare disease with an aggressive clinical course resulting in median survival times of 12 to 14 months.1 It represents approximately 0.8% of primary cutaneous lymphomas.2 According to the World Health Organization Classification of Tumors of Hematopoietic and Lymphoid Tissue, published in 2008, BPDCN is defined as an acute myeloid leukemia related precursor neoplasm, derived from precursors of the plasmacytoid den…

Abstract
Myeloid sarcoma (MS) is a rare neoplastic condition that is often described in association with acute myeloid leukemia (AML). MS in childhood has received little attention, particularly in Japan. We carried out a nationwide retrospective analysis of Japanese children diagnosed with MS without bone marrow involvement. Inclusion criteria were diagnosis of MS at younger than 20 years of age between January 1, 2000 and December 31, 2013. There was a predominance of males (8:2), and the median age at MS diagnosis was 4 years. Sites of involvement varied and included skin (n = 3), head a…

Source: International Journal of HematologyCategory: Hematology Authors: Tags: Int J Hematol Source Type: research

Publication date: Available online 3 May 2018 Source:Oral Surgery, Oral Medicine, Oral Pathology and Oral Radiology Author(s): Fang Yu, Ke Sun, Zhaoming Wang Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare disease with an aggressive clinical course resulting in median survival times of 12-14 months1. It represents approximately 0.8% of primary cutaneous lymphomas2. According to the WHO Classification of Tumors of Hematopoietic and Lymphoid Tissue in 2008, BPDCN is defined as an acute myeloid leukemia related precursor neoplasm, derived from precursors of the plasmacytoid dendritic cell. It is characterized …

Summary
A disease risk index (DRI) has been defined for stratifying heterogeneous cohorts of patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT). This index defines 4 distinct groups with different outcomes, dividing patients by disease type and status and considering cytogenetics for acute myeloid leukaemia and myelodysplastic syndromes (MDS). Recently, the DRI has been refined to include rare diseases and improve MDS stratification by blast percentage and response to prior therapy. Previous reports on DRI include only a small number of UCBT recipients. The current study aims to determine the ap…

Source: British Journal of HaematologyCategory: Hematology Authors: Tags: Research Paper Source Type: research

Beriberi is a rare disease caused by thiamine (vitamin B1) deficiency. There are two types: dry beriberi affecting the neurologic system and wet beriberi leading to high cardiac output heart failure. In extreme cases, brain damage may occur: Wernicke-Korsakoff syndrome. The most common risk factors for thiamine deficiency are: alcohol abuse, overt malnutrition, gastric bypass surgery, total parenteral nutrition (TPN), chronic dialysis, and cancer related cachexia.

Rearrangements of FGFR1 result in the 8p11 myeloproliferative syndrome, a group of rare diseases that features a myeloproliferative neoplasm (MPN) that commonly progresses to lymphoblastic leukemia/lymphoma or acute myeloid leukemia. The most common partner of the FGFR1 is ZMYM2 and patients with the ZMYM2-FGFR1 fusion often present with MPN and T-lymphoblastic lymphoma. There are 14 other partners that can fuse with the FGFR1 and of interest is the BCR-FGFR1 fusion that results from t(8;22)(p11.2;q11.2).

Source: Human PathologyCategory: Pathology Authors: Source Type: research

We present a rare case of so-called aleukemic myeloid leukemia cutis in an 82-year-old man subsequently diagnosed as having MDS without progression to overt AML. This case also illustrates a seemingly rare Kikuchi disease-like inflammatory pattern that obscured the underlying leukemic infiltrate in the skin, delaying its recognition.
PMID: 28376531 [PubMed – in process]

We present a case of MS characterized by systemic lymphadenopathies and multiple effusions, which presumably had a very poor prognosis. The patient was treated with acute myeloid leukemia-type induction chemotherapy and autologous peripheral blood stem cell transplantation, and, unexpectedly, she has remained disease free for more than 6 years. We also reviewed the literature on this rare disease, and found that multiple nonleukemic MS was associated with younger age and a worse prognosis when compared with the overall nonleukemic MS population. We suggest that autologous HCT represents a valid option for young patients wi…

Source: Acta HaematologicaCategory: Hematology Authors: Tags: Acta Haematol Source Type: research

We present a case of MS characterized by systemic lymphadenopathies and multiple effusions, which presumably had a very poor prognosis. The patient was treated with acute myeloid leukemia-type induction chemotherapy and autologous peripheral blood stem cell transplantation, and, unexpectedly, she has remained disease free for more than 6 years. We also reviewed the literature on this rare disease, and found that multiple nonleukemic MS was associated with younger age and a worse prognosis when compared with the overall nonleukemic MS population. We suggest that autologous HCT represents a valid option for young patients wi…

Purpose of review: Mixed-phenotype acute leukemia (MPAL) is a rare disease that poses many diagnostic and therapeutic challenges. Patients with MPAL are considered to have poor outcomes. The difficulties in classifying this leukemia, the lack of prospectively collected data concerning therapeutic outcomes, and rare incidence result in much uncertainty as to the best approach for patients with MPAL.
Recent findings: Recent studies demonstrated that most MPALs are associated with cytogenetic abnormalities; genetic sequencing studies disclose a high frequency of somatic mutations in genes encoding epigenetic regulators, tumo…

Source: Current Opinion in HematologyCategory: Hematology Tags: MYELOID DISEASE: Edited by Martin S. Tallman Source Type: research





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